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NCT01640301 · REGISTRY CONDITION SEARCH

Laboratory-Treated T Cells in Treating Patients With High-Risk Relapsed Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia Previously Treated With Donor Stem Cell Transplant

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE1, PHASE2
Status at capture
TERMINATED
Registry last update
2022-07-14

This phase I/II trial studies the side effects of laboratory-treated T cells and to see how well they work in treating patients with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelogenous leukemia (CML) that has returned after a period of improvement (relapsed), previously treated with donor stem cell transplant. Biological therapies, such as cellular adoptive immunotherapy, may stimulate the immune system in different ways and stop cancer cells from growing. Placing a gene that has been created in the laboratory into a person's T cells may make the body build an immune response to kill cancer cells.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Anti-leukemic Potential Efficacy, in Terms of Duration of Response (Arm II)

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outcome · POSTED

Efficacy, in Terms of Relapse Rate (Arm I)

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outcome · POSTED

Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm I)

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outcome · POSTED

Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm II)

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outcome · POSTED

Count of Participants Who Experienced Grade III-IV Acute Graft Versus Host Disease (GVHD) (Arm II)

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outcome · POSTED

Count of Participants Who Experienced Grade III-IV Acute Graft-versus-host Disease (GVHD) (Arm I)

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outcome · POSTED

Treatment-related Toxicity Rate (Arm I)

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outcome · POSTED

Treatment-related Toxicity Rate (Arm II)

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outcome · POSTED

Disease-free Survival After T Cell Therapy

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outcome · POSTED

Incidence of Relapse After T Cell Therapy (Arm II)

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outcome · POSTED

Maintenance of T Cell Receptor (TCR) Expression of Transduced T Cells (Arm I) i.e. Persistence

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outcome · POSTED

Maintenance of Function of Transduced T Cells (Arm I)

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outcome · POSTED

Time to Progression After T Cell Therapy (Arm I)

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * Patients must express HLA-A\*0201 * Patients who are currently undergoing or who previously underwent matched allogeneic HCT for: * AML: Prospective enrollment will now be limited to patients with relapsed disease (overt relapse or minimal residual disease) at any time post allogeneic HCT * MDS will no longer be a criterion for eligibility * CML will no longer be a criterion for eligibility * Patients must have an HLA-matched donor of hematopoietic stem cells (related or unrelated) * Patients must be able to provide blood and bone marrow samples and undergo the procedures required for this protocol * Patients must be \>= 15 kg, as patients with lower weight would be incapable of providing high volume and frequent blood samples for monitoring and analysis * Patients must be able to give informed consent; parent or legal representative will be asked to consent for patients younger than 18 year old * DONOR: Patient and donor (related or unrelated) must be HLA-matched and express HLA-A\*0201 * DONOR: Donor must be Epstein-Barr virus (EBV) or cytomegalovirus (CMV) seropositive * DONOR: Donor must be age 18 or older * DONOR: In good general health * DONOR: Able to give informed consent Exclusion Criteria: * Central nervous system (CNS) tumor refractory to intrathecal chemotherapy and/or cranio-spinal radiation * In patients whose leukemic cells are available for evaluation, the expression of WT1 in the patient's bone marrow will be determined; if WT1 expression in the patient's bone marrow is not highly expressed by polymerase chain reaction (PCR), the patient will be excluded from the study; patients with no evaluable leukemia will be eligible for enrollment based on the high frequency of positive leukemias (\> 90%), and leukemia will be evaluated for WT1 expression if recurrence is detected * Human immunodeficiency virus (HIV) seropositive; testing for HIV should be within 6 months of enrollment * Medical or psychological conditions that would make the patient unsuitable candidate for cell therapy at the discretion of the principal investigator (PI) * Pregnancy or breast-feeding; women of childbearing potential must have a negative serum or urine beta-human chorionic gonadotropin (B-hCG) pregnancy test result within 14 days before the first dose of WT1-specific T cell infusion; woman of non-childbearing potential will be defined as being postmenopausal greater than one year or who have had a bilateral tubal ligation or hysterectomy; all recipients of WT1-specific T cells will be counseled to use effective birth control during participation in this study and for 12 months after the last T cell infusion * DONOR: Less than 18 years old * DONOR: Active infectious hepatitis * DONOR: HIV or human T-lymphotropic virus (HTLV) seropositive * DONOR: Pregnancy or nursing * DONOR: Significant medical conditions (e.g. immunosuppressive therapy) that would make the donor an unsuitable T cell donor * DONOR: Unable to give informed consent
healthy Volunteers
false
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Patients with no evidence of leukemia or MDS post-HCT receive WT1-sensitized T cells IV over 45 minutes (or longer for patients who are 15-30 kg) on days 0 and 14 and aldesleukin SC BID on days 14-28.
    intervention Names
    1. Biological: Aldesleukin
    2. Other: Laboratory Biomarker Analysis
    3. Biological: WT1-Sensitized Allogeneic T-Lymphocytes
    label
    Arm I (high-risk for relapse after HCT)
    type
    EXPERIMENTAL
  2. description
    Patients with evidence of AML (minimal residual disease or overt relapse) post-HCT receive cyclophosphamide IV and fludarabine phosphate IV daily on days -4 to -2. Patients also receive WT1-sensitized T cells IV over 45 minutes (or longer for patients who are 15-30 kg) on days 0 and 21 and aldesleukin SC BID on days 14-28.
    intervention Names
    1. Biological: Aldesleukin
    2. Drug: Cyclophosphamide
    3. Drug: Fludarabine Phosphate
    4. Other: Laboratory Biomarker Analysis
    5. Biological: WT1-Sensitized Allogeneic T-Lymphocytes
    label
    Arm II (relapsed after HCT)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Arm I (high-risk for relapse after HCT)
    2. Arm II (relapsed after HCT)
    description
    Given SC
    name
    Aldesleukin
    other Names
    1. 125-L-Serine-2-133-interleukin 2
    2. Proleukin
    3. r-serHuIL-2
    4. Recombinant Human IL-2
    5. Recombinant Human Interleukin-2
    type
    BIOLOGICAL
  2. arm Group Labels
    1. Arm II (relapsed after HCT)
    description
    Given IV
    name
    Cyclophosphamide
    other Names
    1. (-)-Cyclophosphamide
    2. 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate
    3. Carloxan
    4. Ciclofosfamida
    5. Ciclofosfamide
    6. Cicloxal
    7. Clafen
    8. Claphene
    9. CP monohydrate
    10. CTX
    11. CYCLO-cell
    12. Cycloblastin
    type
    DRUG
  3. arm Group Labels
    1. Arm II (relapsed after HCT)
    description
    Given IV
    name
    Fludarabine Phosphate
    other Names
    1. 2-F-ara-AMP
    2. 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-
    3. Beneflur
    4. Fludara
    5. SH T 586
    type
    DRUG
  4. arm Group Labels
    1. Arm I (high-risk for relapse after HCT)
    2. Arm II (relapsed after HCT)
    description
    Correlative studies
    name
    Laboratory Biomarker Analysis
    type
    OTHER
  5. arm Group Labels
    1. Arm I (high-risk for relapse after HCT)
    2. Arm II (relapsed after HCT)
    description
    Given IV
    name
    WT1-Sensitized Allogeneic T-Lymphocytes
    type
    BIOLOGICAL
Study design
allocation
NON_RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
47
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Response is assessed throughout the 1 year post treatment timeframe. Morphologic criteria for response: Complete Response (CR) = Bone Marrow blasts \<5%; no blasts with Auer rods; no extramedullary disease. Transfusion independent. Platelets ≥ 100,000/μl Absolute neutrophil count \>1000/μl (CRi: incomplete hematologic recovery CRp: incomplete platelet recovery) Partial Response (PR) = Decrease of at least 50% in the percentage of blasts to 5-25% in the bone marrow and the normalization of blood counts as for CR. (PRi and PRp if incomplete hematologic or platelet recovery) Stable Disease (SD) = Failure to achieve at least PR, but no evidence of progression for \>4 weeks.
    measure
    Anti-leukemic Potential Efficacy, in Terms of Duration of Response (Arm II)
    time Frame
    Up to 1 year
  2. measure
    Efficacy, in Terms of Relapse Rate (Arm I)
    time Frame
    At 1 year post-transplant
  3. measure
    Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm I)
    time Frame
    Up to 1 year
  4. measure
    Count of Participants Who Experienced Chronic Graft Versus Host Disease (GVHD) (Arm II)
    time Frame
    Up to 1 year
  5. measure
    Count of Participants Who Experienced Grade III-IV Acute Graft Versus Host Disease (GVHD) (Arm II)
    time Frame
    Up to 1 year following infusion per patient
  6. measure
    Count of Participants Who Experienced Grade III-IV Acute Graft-versus-host Disease (GVHD) (Arm I)
    time Frame
    Up to 1 year
  7. description
    Outcome will be reported as a count of participants that experienced adverse events. Toxicity will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 4.0
    measure
    Treatment-related Toxicity Rate (Arm I)
    time Frame
    Up to 30 days after last study intervention per patient
  8. description
    Outcome will be reported as a count of participants that experienced adverse events. Toxicity will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 4.0
    measure
    Treatment-related Toxicity Rate (Arm II)
    time Frame
    Up to 30 days after last study intervention per patient
secondary Outcomes
  1. measure
    Disease-free Survival After T Cell Therapy
    time Frame
    Up to 1 year
  2. measure
    Incidence of Relapse After T Cell Therapy (Arm II)
    time Frame
    Up to 1 year
  3. measure
    Maintenance of T Cell Receptor (TCR) Expression of Transduced T Cells (Arm I) i.e. Persistence
    time Frame
    Up to 28 days post intervention per patient
  4. measure
    Maintenance of Function of Transduced T Cells (Arm I)
    time Frame
    Up to 28 days post intervention per patient
  5. measure
    Time to Progression After T Cell Therapy (Arm I)
    time Frame
    Up to 1 year
Full study description
brief Summary
This phase I/II trial studies the side effects of laboratory-treated T cells and to see how well they work in treating patients with high-risk acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelogenous leukemia (CML) that has returned after a period of improvement (relapsed), previously treated with donor stem cell transplant. Biological therapies, such as cellular adoptive immunotherapy, may stimulate the immune system in different ways and stop cancer cells from growing. Placing a gene that has been created in the laboratory into a person's T cells may make the body build an immune response to kill cancer cells.
detailed Description
PRIMARY OBJECTIVES: I. Determine the safety and potential toxicities associated with treating patients with high risk or relapsed AML, MDS, and CML after allogeneic hematopoietic cell transplantation (HCT) by adoptive transfer of virus-specific cluster of differentiation (CD)8 T cells genetically-modified to express a high affinity Wilms tumor 1 (WT1)-specific T cell receptor (TCR). II. Determine the anti-leukemic activity associated with treating patients with relapsed AML, MDS and CML after allogeneic HCT by adoptive transfer of virus-specific CD8 T cells genetically-modified to express a high affinity WT1-specific T cell receptor (TCR). SECONDARY OBJECTIVES: I. Determine the in vivo persistence of transferred T cells and ability to migrate to and accumulate in bone marrow. II. Determine the maintenance of TCR expression and function of transduced T cells. OUTLINE: Patients are assigned to 1 of 2 treatment arms. ARM I: Patients with no evidence of leukemia post-HCT receive WT1-sensitized T cells intravenously (IV) over 45 minutes (or longer for patients who are 15-30 kg) on days 0 and 14 and aldesleukin subcutaneously (SC) twice daily (BID) on days 14-28. ARM II: Patients with evidence of AML (minimal residual disease or overt relapse) post-HCT receive cyclophosphamide IV and fludarabine phosphate IV daily on days -4 to -2. Patients also receive WT1-sensitized T cells IV over 45 minutes (or longer for patients who are 15-30 kg) on days 0 and 21 and aldesleukin SC BID on days 14-28. After completion of study treatment, patients are followed up weekly for 4 weeks, at weeks 6 and 8, at 3, 6, 12 months, and then annually for up to 15 years.
Source references
references
  1. citation
    Chapuis AG, Egan DN, Bar M, Schmitt TM, McAfee MS, Paulson KG, Voillet V, Gottardo R, Ragnarsson GB, Bleakley M, Yeung CC, Muhlhauser P, Nguyen HN, Kropp LA, Castelli L, Wagener F, Hunter D, Lindberg M, Cohen K, Seese A, McElrath MJ, Duerkopp N, Gooley TA, Greenberg PD. T cell receptor gene therapy targeting WT1 prevents acute myeloid leukemia relapse post-transplant. Nat Med. 2019 Jul;25(7):1064-1072. doi: 10.1038/s41591-019-0472-9. Epub 2019 Jun 24.
    pmid
    31235963
    type
    DERIVED
  2. citation
    Oda SK, Daman AW, Garcia NM, Wagener F, Schmitt TM, Tan X, Chapuis AG, Greenberg PD. A CD200R-CD28 fusion protein appropriates an inhibitory signal to enhance T-cell function and therapy of murine leukemia. Blood. 2017 Nov 30;130(22):2410-2419. doi: 10.1182/blood-2017-04-777052. Epub 2017 Oct 17.
    pmid
    29042364
    type
    DERIVED
Source notices and limitations
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    Preserved source evidence · Not medical advice